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What treatments are in research for haemophilia?

What treatments are in research for haemophilia?

Treatments for haemophilia have developed a lot in the past few years. The mainstay of current treatment are clotting factors manufactured without blood donation. Now, a variation of these clotting factors, called extended half-life (EHL) factors are available.50,51,52

The half-life is a measure of how long the clotting factor lasts in your blood stream. EHL factors stay in the blood for longer so people with haemophilia can have the treatment they need to prevent bleeds (prophylaxis) less often.27, 24 With haemophilia A, you may be able to cut down from injections three times a week to twice a week.27 With haemophilia B, you may be able to cut down from twice a week to weekly or even fortnightly injections.24

A treatment that is talked about a lot is gene therapy. This is still in research but is potentially exciting because it could provide a permanent treatment for haemophilia.53 In theory, a single treatment could deliver the gene for making the missing factor and mean that people with haemophilia no longer need factor replacement.53 There are clinical trials underway for gene therapy for both haemophilia A and B.54,55,56

Finally, a new approach to haemophilia treatment is in research. Normally, clotting in the body is held in balance by factors that promote blood clotting and factors that prevent it. Haemophilia researchers have been looking into blocking proteins in the body that normally prevent blood clotting, instead of promoting clotting with injections of factor VIII (8) and factor IX (9). One of these treatments blocks a body protein called anti-thrombin that stops the blood from clotting.57 You have the treatment as an injection under the skin (subcutaneously) once a month. Early research results have shown a drop in bleeding rates and trials are now comparing it to treatment with factor VIII (8) and IX (9).58

Another similar treatment called anti-TFPI is now being tested. TFPIs (tissue factor pathway inhibitors) are proteins that stop the blood clotting and early research has shown that blocking them in people with haemophilia can help the blood to clot.59 Two anti-TFPI treatments in research can be used for either haemophilia A and haemophilia B and are being tested in people with and without inhibitors.60,61,62 As with anti-thrombin, you can have them as injections under the skin (subcutaneously). They are being tested in people who normally have prophylaxis and in those who prefer on-demand treatment.61,62